Phase I Study of Human Von Willebrand Factor for Von Willebrand's Disease
Summary
OBJECTIVES: I. Evaluate the effect of a new von Willebrand factor concentrate on bleeding time, in vivo recovery, and circulating half-life of the infused factor in patients with von Willebrand's disease.
II. Assess the safety of von Willebrand factor in these patients.
Description
PROTOCOL OUTLINE: Patients receive 1 dose of von Willebrand factor concentrate. Timed blood studies are performed for the next 96 hours.
Patients are followed every 2 weeks for 16 weeks, and at 24, 36, and 52 weeks.
Study Design
Endpoint Classification: Safety/Efficacy Study, Primary Purpose: Treatment
Conditions
Von Willebrand's Disease
Intervention
von Willebrand factor
Status
Completed
Source
Office of Rare Diseases (ORD)
Results (where available)
Links
- Source: http://clinicaltrials.gov/show/NCT00004667
- Information obtained from ClinicalTrials.gov on July 15, 2010
Medical and Biotech [MESH] Definitions
Von Willebrand Disease, Type 1
A subtype of von Willebrand disease that results from a partial deficiency of VON WILLEBRAND FACTOR.
Von Willebrand Disease, Type 2
A subtype of von Willebrand disease that results from qualitative deficiencies of VON WILLEBRAND FACTOR. The subtype is divided into several variants with each variant having a distinctive pattern of PLATELET-interaction.
Von Willebrand Disease, Type 3
A subtype of von Willebrand disease that results from a total or near total deficiency of VON WILLEBRAND FACTOR.
Von Willebrand Factor
A high-molecular-weight plasma protein, produced by endothelial cells and megakaryocytes, that is part of the factor VIII/von Willebrand factor complex. The von Willebrand factor has receptors for collagen, platelets, and ristocetin activity as well as the immunologically distinct antigenic determinants. It functions in adhesion of platelets to collagen and hemostatic plug formation. The prolonged bleeding time in VON WILLEBRAND DISEASES is due to the deficiency of this factor.
Von Willebrand Diseases
Group of hemorrhagic disorders in which the VON WILLEBRAND FACTOR is either quantitatively or qualitatively abnormal. They are usually inherited as an autosomal dominant trait though rare kindreds are autosomal recessive. Symptoms vary depending on severity and disease type but may include prolonged bleeding time, deficiency of factor VIII, and impaired platelet adhesion.
Clinical Trials
The objectives of this study are to evaluate the immediate tolerability and safety of rVWF:rFVIII in subjects with Type 3 Von Willebrand Disease after administration of various dosages of...
National Study of Moderate and Severe Von Willebrand Disease in the Netherlands
The objective of this study is to assess the clinical presentation, the treatment and the complications of the disease and treatment in moderate and severe von Willebrand disease. Another...
Collection of Blood in Normal Subjects and Subjects With Von Willebrand Disease (VWD)
Von Willebrand disease is an inherited bleeding disorder that impacts the blood's ability to clot properly. Von Willebrand disease is cause by the lack or not working substance in the bloo...
Phase II Study of IL-11 (Neumega) in Von Willebrand Disease
This study is testing the use of rhIL-11 (recombinant interleukin 11, Neumega) in individuals with Von Willebrand disease. The purpose is to evaluate: 1. if rhIL-11 corrects VWF (Von Wi...
The Von Willebrand Disease (VWD) International Prophylaxis Study
The von Willebrand Disease Prophylaxis Network (vWD PN) is an international study group formed with the goal of investigating the role of prophylaxis in clinically severe VWD that is non-r...
PubMed Articles
Principles of care for the diagnosis and treatment of von Willebrand disease.
Von Willebrand disease is a common autosomal inherited bleeding disorder caused by quantitative or qualitative defects of von Willebrand factor, a multi-adhesive protein that binds platelets to expose...
We have studied the effect of a 13 bp deletion in the promoter of the von Willebrand factor (VWF) gene in a patient with type 1 von Willebrand disease (VWD). The index case has a VWF:Ag of 0.49 IU/ mL...
No excess surgical blood loss in patients with acquired type 2A von Willebrand disease.
Abstract Objectives. Acquired type 2A von Willebrand disease may develop as a consequence of aortic valve stenosis and is associated with varying degrees of bleeding tendency. It remains unknown, whet...
Evaluation of an Automated von Willebrand Factor Activity Assay in von Willebrand Disease.
We evaluated the use of the turbidimetric HemosIL von Willebrand Factor (VWF) Activity assay (VWF:Act) on the STA-R automated coagulometer (Stago, Asnières, France) for the diagnosis of von Willebran...
Type 3 von Willebrand's disease (VWD) is a rare bleeding diathesis with complete or near complete deficiency of von Willebrand factor (VWF) and low factor VIII (FVIII) levels. In contrast, only FVIII...
